Government Shutdown Impacts FDA Drug Approvals
Now in its fourth week, the longest shutdown in the history of the federal government is having an impact on patient care, as the US Food and Drug Administration (FDA) has put drug reviews and approvals for new drugs and devices, as well as the issuance of new guidance documents, on indefinite hold. The FDA recently posted on its website a notice alerting the public of its current operational status.
While about 75% of federal agencies had secured FY2019 funding prior to the political impasse, the FDA had not, and is among the agencies that faced partial shutdown. Approximately 40% of the FDAâ€™s entire workforce has been furloughed since December 22, 2018, when the government stopped operations.
Some vital public safety functions continue, including inspection of imported drugs, but this has diverted personnel from tasks considered nonessential. Several pharmaceutical companies that had hoped for early 2019 approvals now hang in suspense, with some inside the government speculating that the political crisis may not be resolved for weeks to come. As a result of heavy backlogs, prescribers and patients will likely face extended wait times for approvals even after the restoration of the agency to its full staffing once the stalemate is broken and government business resumes.
Under the Prescription Drug User Fee Act, the FDA uses the fees collected from drug manufacturers to fund drug reviews and approvals and is currently using leftover monies to finish working on applications submitted in FY2018; however, that money is expected to run out in early February. The FDA is accepting no new testing applications or fees, and in addition, is reallocating the funds previously collected for premarket review work to prioritize postmarket safety surveillance, according to the FDA Commissioner Scott Gottlieb. With no end to the shutdown in sight, the long-term consequences on the pharmaceutical industry and patient care remain an open question.
FDA Will Require Early Assessment of New Targeted Agents That May Be Used in Pediatric Cancers
On December 12, 2019, the FDA issued draft guidance to implement amendments to the Federal Food, Drug, and Cosmetic (FD&C) Act that will facilitate early assessment of studies of molecularly targeted oncology drugs that may be effective in the treatment of pediatric cancers.
FDA Grants Approval to Avapritinib for Patients with GIST
On January 9, 2020, the FDA approved avapritinib (Ayvakit; Blueprint Medicines), a kinase inhibitor, for the treatment of adult patients with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutations, including PDGFRA D842V mutations.